JP6975641B2 - アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法 - Google Patents

アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法 Download PDF

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JP6975641B2
JP6975641B2 JP2017553347A JP2017553347A JP6975641B2 JP 6975641 B2 JP6975641 B2 JP 6975641B2 JP 2017553347 A JP2017553347 A JP 2017553347A JP 2017553347 A JP2017553347 A JP 2017553347A JP 6975641 B2 JP6975641 B2 JP 6975641B2
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angptl3
dsrna
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irna
expression
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JP2018512155A (ja
JP2018512155A5 (cg-RX-API-DMAC7.html
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ウィリアム・クアーベス
ケビン・フィッツジェラルド
ジェイムズ・バトラー
ステファニー・ウィリアムズ
グレゴリー・ヒンクル
マルティン・マイアー
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Alnylam Pharmaceuticals Inc
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JP2017553347A 2015-04-13 2016-04-13 アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法 Active JP6975641B2 (ja)

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JP2021147698A JP2022002518A (ja) 2015-04-13 2021-09-10 アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法
JP2023169168A JP2024009838A (ja) 2015-04-13 2023-09-29 アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法

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US201562146604P 2015-04-13 2015-04-13
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US201562261361P 2015-12-01 2015-12-01
US62/261,361 2015-12-01
PCT/US2016/027271 WO2016168286A1 (en) 2015-04-13 2016-04-13 Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof

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JP2023169168A Pending JP2024009838A (ja) 2015-04-13 2023-09-29 アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法

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Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
TWI862799B (zh) 2015-04-13 2024-11-21 美商阿尼拉製藥公司 類血管生成素3(ANGPTL3)iRNA組成物及其用途方法
MX2020001912A (es) * 2017-09-14 2020-03-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso.
JP7273417B2 (ja) 2017-12-01 2023-05-15 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用
JP7727308B2 (ja) * 2017-12-01 2025-08-21 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 二本鎖オリゴヌクレオチド、二本鎖オリゴヌクレオチドを含む組成物および複合体ならびに調製方法と使用
US12083142B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
DK3732185T3 (da) * 2017-12-29 2025-03-31 Suzhou Ribo Life Science Co Ltd Konjugater og fremstilling og anvendelse heraf
TWI851574B (zh) * 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
US20210388356A1 (en) * 2018-11-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotide
WO2020099482A2 (en) * 2018-11-13 2020-05-22 Lipigon Pharmaceuticals Ab Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism
EP3903830A4 (en) 2018-12-28 2023-04-26 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH NUCLEIC ACID, PROCESS FOR THEIR PREPARATION AND ITS USE
AU2020280438B2 (en) 2019-05-22 2025-03-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
WO2021119019A1 (en) * 2019-12-09 2021-06-17 Empirico Inc. Oligonucleotides for treatment of angiopoietin like 4 (angptl4) related diseases
KR20230011913A (ko) * 2020-03-04 2023-01-25 버브 테라퓨틱스, 인크. 표적화된 rna 전달을 위한 조성물 및 방법
WO2021204872A1 (en) * 2020-04-07 2021-10-14 Uniqure Ip B.V. Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof
EP4223875A1 (en) * 2020-09-30 2023-08-09 Nanopeptide (Qingdao) Biotechnology Ltd. Sirna of angiopoietin-like 3 (angptl3) and use thereof
EP4229200A1 (en) * 2020-10-16 2023-08-23 Sanofi Novel rna compositions and methods for inhibiting angptl3
CN114685585B (zh) * 2020-12-31 2024-06-04 苏州瑞博生物技术股份有限公司 核苷酸序列、双链寡核苷酸、药物组合物与缀合物及制备方法和用途
IL305414A (en) * 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
EP4313075A4 (en) * 2021-03-24 2025-06-18 Atalanta Therapeutics, Inc. DOUBLE-STRANDED SIRNA WITH PATTERNED CHEMICAL MODIFICATIONS
JP2024535852A (ja) * 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ, インコーポレイテッド ANGPTL4をサイレンシングするためのiRNA組成物および方法
KR20240053627A (ko) * 2021-09-23 2024-04-24 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 안지오포이에틴-유사 3(angptl3) 단백질의 발현을 억제하기 위한 조성물 및 방법
WO2023092102A1 (en) * 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
MX2024007900A (es) 2021-12-22 2024-07-10 Regeneron Pharma Tratamiento de enfermedades renales con inhibidores de la angiopoyetina tipo 3 (angptl3).
WO2023134705A1 (zh) * 2022-01-11 2023-07-20 上海金中锘美生物医药科技有限公司 抑制angptl3表达的rna干扰剂及其用途
CN116987696A (zh) * 2022-08-04 2023-11-03 北京福元医药股份有限公司 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024032608A1 (zh) * 2022-08-08 2024-02-15 大睿生物医药科技(上海)有限公司 调控ANGPTL3基因活性的siRNA分子
AU2023375521A1 (en) 2022-11-10 2025-05-15 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with a combination of angiopoietin like 3 (angptl3) inhibitors and solute carrier family 5 member 2 (slc5a2) inhibitors
WO2024227059A2 (en) * 2023-04-27 2024-10-31 Aligos Therapeutics, Inc. Short interfering nucleic acid (sina) molecules targeting angptl 3 and angptl 8 and methods of using the same
TW202530409A (zh) * 2023-10-07 2025-08-01 大陸商齊魯製藥有限公司 iRNA組合物及其使用方法
WO2025169133A1 (en) 2024-02-09 2025-08-14 Astrazeneca Ab Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof
WO2025201277A1 (en) * 2024-03-25 2025-10-02 Shanghai Argo Biopharmaceutical Co., Ltd. Method for treating dyslipidemia with angiopoietin-like 3 (angptl3) -targeted rnai agents

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5783701A (en) 1992-09-08 1998-07-21 Vertex Pharmaceuticals, Incorporated Sulfonamide inhibitors of aspartyl protease
RU2123492C1 (ru) 1993-02-19 1998-12-20 Ниппон Синяку Ко., Лтд Производные глицерина, средство для доставки физиологически активного вещества и фармацевтическая композиция
US6191105B1 (en) 1993-05-10 2001-02-20 Protein Delivery, Inc. Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
US5858401A (en) 1996-01-22 1999-01-12 Sidmak Laboratories, Inc. Pharmaceutical composition for cyclosporines
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
EP1042280A2 (en) 1997-12-24 2000-10-11 Vertex Pharmaceuticals Incorporated Prodrugs of aspartyl protease inhibitors
BR9914773A (pt) 1998-10-09 2002-02-05 Ingene Inc Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica
IL142490A0 (en) 1998-10-09 2002-03-10 Ingene Inc ENZYMATIC SYNTHESIS OF ssDNA
TWI260322B (en) 1999-02-12 2006-08-21 Vertex Pharma Inhibitors of aspartyl protease
IT1318539B1 (it) 2000-05-26 2003-08-27 Italfarmaco Spa Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente
EP1873259B1 (en) 2000-12-01 2012-01-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediated by 21 and 22nt RNAs
US7063860B2 (en) 2001-08-13 2006-06-20 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
EP2314691A3 (en) 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional and hyperfunctional siRNA
ATE541928T1 (de) 2005-03-31 2012-02-15 Calando Pharmaceuticals Inc Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
ZA200904022B (en) 2006-12-08 2010-08-25 Lexicon Pharmaceuticals Inc Monoclonal antibodies against ANGPTL3
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
EP2245039A4 (en) 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
ES2740129T3 (es) 2008-09-25 2020-02-05 Alnylam Pharmaceuticals Inc Composiciones formuladas en lípidos y métodos de inhibición de la expresión del gen de suero amiloide A
JP5791505B2 (ja) 2008-10-20 2015-10-07 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. トランスチレチンの発現を阻害するための組成物および方法
WO2010068816A1 (en) 2008-12-10 2010-06-17 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
EP2380108A4 (en) * 2009-01-20 2013-01-23 Videojet Technologies Inc DATE PRINTING PROCESS
PL2440183T3 (pl) 2009-06-10 2019-01-31 Arbutus Biopharma Corporation Ulepszona formulacja lipidowa
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US9512164B2 (en) 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
WO2011085271A2 (en) * 2010-01-08 2011-07-14 Isis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
US8481292B2 (en) * 2010-09-21 2013-07-09 Population Genetics Technologies Litd. Increasing confidence of allele calls with molecular counting
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
IL303831A (en) * 2011-11-18 2023-08-01 Alnylam Pharmaceuticals Inc Adapted RNAI factors
WO2013165816A2 (en) * 2012-05-02 2013-11-07 Merck Sharp & Dohme Corp. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
AU2013296321B2 (en) 2012-08-03 2019-05-16 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
CA2879693A1 (en) 2012-08-06 2014-02-13 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated rna agents and process for their preparation
TW201515650A (zh) * 2013-05-06 2015-05-01 艾爾妮蘭製藥公司 遞送經脂質調配之核酸分子之劑量及方法
DK3087183T3 (da) 2013-12-24 2020-09-14 Ionis Pharmaceuticals Inc Modulation af angiopoietin-3-lignende ekspression
US9382540B2 (en) * 2014-05-01 2016-07-05 Isis Pharmaceuticals, Inc Compositions and methods for modulating angiopoietin-like 3 expression
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US20180245077A1 (en) 2015-03-20 2018-08-30 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
TWI862799B (zh) 2015-04-13 2024-11-21 美商阿尼拉製藥公司 類血管生成素3(ANGPTL3)iRNA組成物及其用途方法
MX2020001912A (es) 2017-09-14 2020-03-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso.
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
UY38003A (es) 2017-12-12 2019-06-28 Amgen Inc Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas
JOP20210142A1 (ar) 2018-12-10 2023-01-30 Amgen Inc تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
AU2021236674B2 (en) 2020-03-18 2025-03-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting ANGPTL3 expression
US20230357768A1 (en) 2020-09-17 2023-11-09 Astrazeneca Ab Pharmaceutical compositions comprising an antisense oligonucleotide for oral administration
IL305414A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
AU2022298603A1 (en) 2021-06-21 2024-02-01 Shanghai Junshi Biosciences Co., Ltd. Sirna inhibiting angptl3 gene expression, and use thereof
CN120112639A (zh) 2022-08-22 2025-06-06 箭头药业股份有限公司 Angptl3相关疾病和病症的治疗方法
WO2024164008A1 (en) 2023-02-03 2024-08-08 Sirnaomics, Inc. Products and compositions
US20250051771A1 (en) 2023-07-24 2025-02-13 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof

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